Trend WatchPerspective

FDA Approves DAAs for Pediatric Patients with HCV

The FDA has approved sofosbuvir and ledipasvir/sofosbuvir as supplemental applications for the treatment of hepatitis C in children aged 12 to 17 years, according to an FDA news release.

“These approvals will help change the landscape for HCV treatment by addressing an unmet need in children and adolescents,” Edward Cox, MD, director of the office of antimicrobial products in the FDA’s Center for Drug Evaluation and Research, said in the release.

Sofosbuvir (Sovaldi, Gilead) in combination with ribavirin is indicated for the treatment of pediatric patients 12 years and older or at least 77 lb with HCV genotype 2 or 3 without cirrhosis or with mild cirrhosis.

The safety and efficacy of sofosbuvir in combination with ribavirin for pediatric patients was evaluated in an open-label clinical trial of 50 patients 12 years or older. Results were comparable to those observed in adults. At 12 weeks, all patients with genotype 2 and 97% of patients with genotype 3 had no virus detection. The most common adverse events were fatigue and headache.

Ledipasvir (Harvoni/sofosbuvir, Gilead) is indicated for the treatment of pediatric patients 12 years and older or at least 77 lb with HCV genotypes 1, 4, 5 or 6 without cirrhosis or with mild cirrhosis.

The safety and efficacy of ledipasvir/sofosbuvir for pediatric patients with genotype 1 was evaluated in an open-label clinical trial of 100 patients 12 years and older. Results were comparable to those observed in adults. At 12 weeks, 98% of patients had no virus detection. Treatment for genotypes 4, 5 and 6 showed similar safety and efficacy profiles.

According to the FDA, because hepatitis B reactivation has been reported in HCV/HBV coinfected adult patients who underwent or had completed treatment with direct-acting antivirals, all patients, including pediatric patients, should be screened for evidence of current or prior HBV.


Michael S. Saag

  • As the options for treating hepatitis C have expanded dramatically over the last 3 years for adult patients, news this week indicate that the benefits of hepatitis C therapy are now extending into pediatric populations. Sofosbuvir and the combination ledipasvir/sofosbuvir is now approved for children aged between 12 and 17 years who have chronic HCV infection.

    While this population comprises only a small fraction of the number of people infected with HCV in the United States, it does represent between 25,000 and 40,000 children who will benefit from the approval of these two therapeutic options. ...

    In clinical trials, the activity of sofosbuvir combined with ledipasvir or other agents was able to eradicated HCV in up to 98% of children, mirroring the findings in adults. Moreover, the pharmacokinetic profiles indicated that these drugs will be safe in this younger population of patients.

    While it is not a surprise that these drugs are now available for pediatric populations, it is welcome news.

    Owing to the need for the regulatory agency to prove not only efficacy, but also safety in younger populations, it is very common for drug approvals to be delayed, often by a period of years relative to the adult population. Hopefully, several of the other HCV drugs will be approved for this younger population and, ultimately, for children aged younger than 12.

    We eagerly await clinical studies that can document safety and efficacy in these younger populations as well.

    • Michael S. Saag, MD
    • Co-Chief Medical Editor HCV Next
  • Disclosures: Disclosure: Saag reports no relevant financial disclosures.
Philip Rosenthal

Philip Rosenthal

  • This approval is a great advancement. To have the opportunity to treat pediatric patients with these drugs changes the landscape of therapy for HCV infection.

    I was involved in both of the studies and I personally saw 100% success using these drugs. They are safe and effective. In the trials, the only patients whose infection was not eradicated were those who failed to comply with the regimen.

    It is a major breakthrough for children to have oral medications. Previously, pediatric patients could only receive these drugs through clinical trials. Now that these drugs are approved, insurance should cover access to them and children will not have to rely on shots. The short duration of therapy is also key in ensuring effective eradication of the infection.

    They work. Patients are happy, parents are happy and physicians are happy. This approval will lead to a significant increase in availability that will, in turn, mean fewer infected pediatric patients.

    • Philip Rosenthal, MD
    • Director of Pediatric Hepatology University of California, San Francisco
  • Disclosures: Disclosure: Rosenthal reports he receives research support and is a consultant for Gilead.